Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera

Yan Beauverd; Jean-Christophe Ianotto; Kyaw Htin Thaw; Marta Sobas; Parvis Sadjadian; Natalia Curto-Garcia; Lee-Yung Shih; Timothy Devos; Dorota Krochmalczyk; Serena Galli; Maria Bieniaszewska; Ilona Seferynska; Mary Frances McMullin; Anna Armatys; Adrianna Spalek; Joanna Waclaw; Mihnea Tudor Zdrenghea; Laurence Legros; Francois Girodon; Krzysztof Lewandowski; Beatriz Bellosillo; Jan Samuelsson; Aitor Abuin Blanco; Pascale Cony-Makhoul; Angela Collins; Chloe James; Rajko Kusec; Marie Lauermannova; Maria Soledad Noya; Malgorzata Skowronek; Lukasz Szukalski; Anna Szmigielska-Kaplon; Marielle Wondergem; Iryna Dudchenko; Joanna Gora-Tybor; Kamel Laribi; Anna Kulikowska de Nałęcz; Jean-Loup Demory; Katell Le Dû; Sonja Zweegman; Carlos Besses Raebel; Radek C Skoda; Stephane Giraudier; Martin Griesshammer; Jean-Jacques Kiladjian; Claire N Harrison

doi:10.1038/s41375-025-02545-2

Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera

Yan Beauverd, Jean-Christophe Ianotto, Kyaw Htin Thaw, Marta Sobas, Parvis Sadjadian, Natalia Curto-Garcia, Lee-Yung Shih, Timothy Devos, Dorota Krochmalczyk, Serena Galli, Maria Bieniaszewska, Ilona Seferynska, Mary Frances McMullin, Anna Armatys, Adrianna Spalek, Joanna Waclaw, Mihnea Tudor Zdrenghea, Laurence Legros, Francois Girodon, Krzysztof LewandowskiBeatriz Bellosillo, Jan Samuelsson, Aitor Abuin Blanco, Pascale Cony-Makhoul, Angela Collins, Chloe James, Rajko Kusec, Marie Lauermannova, Maria Soledad Noya, Malgorzata Skowronek, Lukasz Szukalski, Anna Szmigielska-Kaplon, Marielle Wondergem, Iryna Dudchenko, Joanna Gora-Tybor, Kamel Laribi, Anna Kulikowska de Nałęcz, Jean-Loup Demory, Katell Le Dû, Sonja Zweegman, Carlos Besses Raebel, Radek C Skoda, Stephane Giraudier, Martin Griesshammer, Jean-Jacques Kiladjian, Claire N Harrison

School of Medicine, Dentistry and Biomedical Sciences

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Abstract

Essential thrombocythemia (ET) and polycythemia vera (PV) are rare in adolescent and young adult (AYA). These conditions, similar to those in older patients, are linked with thrombotic complications and the potential progression to secondary myelofibrosis (sMF). This retrospective study of ET and PV patients diagnosed before age 25 evaluated complication rates and impact of cytoreductive drugs on outcomes. Among 348 patients (278 ET, 70 PV) with a median age of 20 years, the of thrombotic events was 1.9 per 100 patient-years. Risk factors for thrombosis included elevated white blood cell count (>11 × 10 9/L) (HR: 2.7, p = 0.012) and absence of splenomegaly at diagnosis (HR: 5.7, p = 0.026), while cytoreductive drugs did not reduce this risk. The incidence of sMF was 0.7 per 100 patient-years. CALR mutation (HR: 6.0, p < 0.001) and a history of thrombosis (HR: 3.8, p = 0.015) were associated with sMF risk. Interferon as a first-line treatment significantly improved myelofibrosis-free survival compared to other treatments or the absence of cytoreduction (p = 0.046). Although cytoreduction did not affect thrombotic event, early interferon use reduced sMF risk. These findings support interferon use to mitigate sMF risk in AYA ET and PV patients.

Original language	English
Journal	Leukemia
Early online date	12 Mar 2025
DOIs	https://doi.org/10.1038/s41375-025-02545-2
Publication status	Early online date - 12 Mar 2025

Bibliographical note

Keywords

adolescent
essential thrombocythemia
polycythemia vera
Essential thrombocythemia

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Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera
Copyright 2025 the authors. This is an open access article published under a Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution and reproduction in any medium, provided the author and source are cited.
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Beauverd, Y., Ianotto, J.-C., Thaw, K. H., Sobas, M., Sadjadian, P., Curto-Garcia, N., Shih, L.-Y., Devos, T., Krochmalczyk, D., Galli, S., Bieniaszewska, M., Seferynska, I., McMullin, M. F., Armatys, A., Spalek, A., Waclaw, J., Zdrenghea, M. T., Legros, L., Girodon, F., ... Harrison, C. N. (2025). Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera. Leukemia. Advance online publication. https://doi.org/10.1038/s41375-025-02545-2

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title = "Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera",

abstract = "Essential thrombocythemia (ET) and polycythemia vera (PV) are rare in adolescent and young adult (AYA). These conditions, similar to those in older patients, are linked with thrombotic complications and the potential progression to secondary myelofibrosis (sMF). This retrospective study of ET and PV patients diagnosed before age 25 evaluated complication rates and impact of cytoreductive drugs on outcomes. Among 348 patients (278 ET, 70 PV) with a median age of 20 years, the of thrombotic events was 1.9 per 100 patient-years. Risk factors for thrombosis included elevated white blood cell count (>11 × 10 9/L) (HR: 2.7, p = 0.012) and absence of splenomegaly at diagnosis (HR: 5.7, p = 0.026), while cytoreductive drugs did not reduce this risk. The incidence of sMF was 0.7 per 100 patient-years. CALR mutation (HR: 6.0, p < 0.001) and a history of thrombosis (HR: 3.8, p = 0.015) were associated with sMF risk. Interferon as a first-line treatment significantly improved myelofibrosis-free survival compared to other treatments or the absence of cytoreduction (p = 0.046). Although cytoreduction did not affect thrombotic event, early interferon use reduced sMF risk. These findings support interferon use to mitigate sMF risk in AYA ET and PV patients. ",

keywords = "adolescent, essential thrombocythemia, polycythemia vera, Essential thrombocythemia",

author = "Yan Beauverd and Jean-Christophe Ianotto and Thaw, \{Kyaw Htin\} and Marta Sobas and Parvis Sadjadian and Natalia Curto-Garcia and Lee-Yung Shih and Timothy Devos and Dorota Krochmalczyk and Serena Galli and Maria Bieniaszewska and Ilona Seferynska and McMullin, \{Mary Frances\} and Anna Armatys and Adrianna Spalek and Joanna Waclaw and Zdrenghea, \{Mihnea Tudor\} and Laurence Legros and Francois Girodon and Krzysztof Lewandowski and Beatriz Bellosillo and Jan Samuelsson and \{Abuin Blanco\}, Aitor and Pascale Cony-Makhoul and Angela Collins and Chloe James and Rajko Kusec and Marie Lauermannova and Noya, \{Maria Soledad\} and Malgorzata Skowronek and Lukasz Szukalski and Anna Szmigielska-Kaplon and Marielle Wondergem and Iryna Dudchenko and Joanna Gora-Tybor and Kamel Laribi and \{Kulikowska de Na{\l}{\c e}cz\}, Anna and Jean-Loup Demory and \{Le D{\^u}\}, Katell and Sonja Zweegman and \{Besses Raebel\}, Carlos and Skoda, \{Radek C\} and Stephane Giraudier and Martin Griesshammer and Jean-Jacques Kiladjian and Harrison, \{Claire N\}",

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year = "2025",

month = mar,

day = "12",

doi = "10.1038/s41375-025-02545-2",

language = "English",

journal = "Leukemia",

issn = "0887-6924",

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Beauverd, Y, Ianotto, J-C, Thaw, KH, Sobas, M, Sadjadian, P, Curto-Garcia, N, Shih, L-Y, Devos, T, Krochmalczyk, D, Galli, S, Bieniaszewska, M, Seferynska, I, McMullin, MF, Armatys, A, Spalek, A, Waclaw, J, Zdrenghea, MT, Legros, L, Girodon, F, Lewandowski, K, Bellosillo, B, Samuelsson, J, Abuin Blanco, A, Cony-Makhoul, P, Collins, A, James, C, Kusec, R, Lauermannova, M, Noya, MS, Skowronek, M, Szukalski, L, Szmigielska-Kaplon, A, Wondergem, M, Dudchenko, I, Gora-Tybor, J, Laribi, K, Kulikowska de Nałęcz, A, Demory, J-L, Le Dû, K, Zweegman, S, Besses Raebel, C, Skoda, RC, Giraudier, S, Griesshammer, M, Kiladjian, J-J & Harrison, CN 2025, 'Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera', Leukemia. https://doi.org/10.1038/s41375-025-02545-2

TY - JOUR

T1 - Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera

AU - Beauverd, Yan

AU - Ianotto, Jean-Christophe

AU - Thaw, Kyaw Htin

AU - Sobas, Marta

AU - Sadjadian, Parvis

AU - Curto-Garcia, Natalia

AU - Shih, Lee-Yung

AU - Devos, Timothy

AU - Krochmalczyk, Dorota

AU - Galli, Serena

AU - Bieniaszewska, Maria

AU - Seferynska, Ilona

AU - McMullin, Mary Frances

AU - Armatys, Anna

AU - Spalek, Adrianna

AU - Waclaw, Joanna

AU - Zdrenghea, Mihnea Tudor

AU - Legros, Laurence

AU - Girodon, Francois

AU - Lewandowski, Krzysztof

AU - Bellosillo, Beatriz

AU - Samuelsson, Jan

AU - Abuin Blanco, Aitor

AU - Cony-Makhoul, Pascale

AU - Collins, Angela

AU - James, Chloe

AU - Kusec, Rajko

AU - Lauermannova, Marie

AU - Noya, Maria Soledad

AU - Skowronek, Malgorzata

AU - Szukalski, Lukasz

AU - Szmigielska-Kaplon, Anna

AU - Wondergem, Marielle

AU - Dudchenko, Iryna

AU - Gora-Tybor, Joanna

AU - Laribi, Kamel

AU - Kulikowska de Nałęcz, Anna

AU - Demory, Jean-Loup

AU - Le Dû, Katell

AU - Zweegman, Sonja

AU - Besses Raebel, Carlos

AU - Skoda, Radek C

AU - Giraudier, Stephane

AU - Griesshammer, Martin

AU - Kiladjian, Jean-Jacques

AU - Harrison, Claire N

PY - 2025/3/12

Y1 - 2025/3/12

N2 - Essential thrombocythemia (ET) and polycythemia vera (PV) are rare in adolescent and young adult (AYA). These conditions, similar to those in older patients, are linked with thrombotic complications and the potential progression to secondary myelofibrosis (sMF). This retrospective study of ET and PV patients diagnosed before age 25 evaluated complication rates and impact of cytoreductive drugs on outcomes. Among 348 patients (278 ET, 70 PV) with a median age of 20 years, the of thrombotic events was 1.9 per 100 patient-years. Risk factors for thrombosis included elevated white blood cell count (>11 × 10 9/L) (HR: 2.7, p = 0.012) and absence of splenomegaly at diagnosis (HR: 5.7, p = 0.026), while cytoreductive drugs did not reduce this risk. The incidence of sMF was 0.7 per 100 patient-years. CALR mutation (HR: 6.0, p < 0.001) and a history of thrombosis (HR: 3.8, p = 0.015) were associated with sMF risk. Interferon as a first-line treatment significantly improved myelofibrosis-free survival compared to other treatments or the absence of cytoreduction (p = 0.046). Although cytoreduction did not affect thrombotic event, early interferon use reduced sMF risk. These findings support interferon use to mitigate sMF risk in AYA ET and PV patients.

AB - Essential thrombocythemia (ET) and polycythemia vera (PV) are rare in adolescent and young adult (AYA). These conditions, similar to those in older patients, are linked with thrombotic complications and the potential progression to secondary myelofibrosis (sMF). This retrospective study of ET and PV patients diagnosed before age 25 evaluated complication rates and impact of cytoreductive drugs on outcomes. Among 348 patients (278 ET, 70 PV) with a median age of 20 years, the of thrombotic events was 1.9 per 100 patient-years. Risk factors for thrombosis included elevated white blood cell count (>11 × 10 9/L) (HR: 2.7, p = 0.012) and absence of splenomegaly at diagnosis (HR: 5.7, p = 0.026), while cytoreductive drugs did not reduce this risk. The incidence of sMF was 0.7 per 100 patient-years. CALR mutation (HR: 6.0, p < 0.001) and a history of thrombosis (HR: 3.8, p = 0.015) were associated with sMF risk. Interferon as a first-line treatment significantly improved myelofibrosis-free survival compared to other treatments or the absence of cytoreduction (p = 0.046). Although cytoreduction did not affect thrombotic event, early interferon use reduced sMF risk. These findings support interferon use to mitigate sMF risk in AYA ET and PV patients.

KW - adolescent

KW - essential thrombocythemia

KW - polycythemia vera

KW - Essential thrombocythemia

U2 - 10.1038/s41375-025-02545-2

DO - 10.1038/s41375-025-02545-2

M3 - Article

C2 - 40074852

SN - 0887-6924

JO - Leukemia

JF - Leukemia

ER -

Impact of treatment for adolescent and young adults with essential thrombocythemia and polycythemia vera

Abstract

Bibliographical note

Keywords

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